FDA Approves Merck’s Welireg for Cancers Linked With Rare Genetic Disease

Merck has won FDA approval for Welireg (belzutifan) for treating adults with cancers associated with von Hippel-Lindau (VHL) disease, a rare genetic disorder that causes tumors.

Welireg, which works by reducing expression of certain genes involved in the proliferation of cancer cells and tumors, showed “an overall response rate across three different types of VHL-associated tumors” in a clinical trial, according to Eric Jonasch, the study’s principal investigator.

Merck’s application was evaluated under the FDA’s Project Orbis in which oncology drugs are reviewed concurrently by regulatory authorities in several countries. For this review, the FDA collaborated with the Australian Therapeutic Goods Administration, Health Canada and the UK’s Medicines and Healthcare products Regulatory Agency.